PR Newswire Clinical TrialsOriginal article
Ractigen Therapeutics Presents Positive First-in-Human Data for RAG-18 at WMS 2026, Establishing Clinical Proof-of-Mechanism for RNA Activation in Duchenne Muscular Dystrophy
Phase 1positiveRAG-18Positive
AI Analysis
Summary
Ractigen Therapeutics presented positive first-in-human clinical data for RAG-18, demonstrating that a systemically delivered small activating RNA can safely upregulate endogenous utrophin expression in human skeletal muscle for Duchenne Muscular Dystrophy treatment.
Clinical Trial Data
Phase
Phase 1
Primary Endpoint
Met
Outcome Details
First-in-human data demonstrated safe systemic delivery of small activating RNA (saRNA) with 3.5- to 5.3-fold increases in sarcolemmal utrophin expression in human skeletal muscle
Importance:7/10
Sentiment:
0.85
RNA ActivationMuscular DystrophyGene TherapyClinical Proof-of-MechanismFirst-in-HumanWMS 2026 Conference
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Published by PR Newswire Clinical Trials on October 3, 2026 11:30 AM